Alnylam Pharmaceuticals

Vir Biotechnology announced FDA Breakthrough Therapy and EMA PRIME designations for tobevibart and elebsiran in chronic hepatitis delta, supported by Phase 2 SOLSTICE trial data. The Phase 3 ECLIPSE program will start in H1 2025.

Vir Biotechnology's tobevibart and elebsiran received FDA Breakthrough Therapy and EMA PRIME designations for chronic hepatitis delta, based on positive Phase 2 SOLSTICE trial data. The company plans to start Phase 3 ECLIPSE program in 2025.

Vir Biotechnology announced that tobevibart and elebsiran received FDA Breakthrough Therapy and EMA PRIME designations for treating chronic hepatitis delta (CHD), supported by Phase 2 SOLSTICE trial data. The Phase 3 ECLIPSE program will start in H1 2025.

Regeneron achieved significant trial results at the American Society for Hematology meeting, demonstrating better disease control in paroxysmal nocturnal hemoglobinuria with a combination therapy and potential best-in-class performance in follicular lymphoma with a bispecific antibody. Dr. Andres Sirulnik highlights Regeneron's science-driven approach and the company's focus on addressing difficult-to-treat problems in hematology.

uniQure's QURE surged 109.7% after aligning with FDA on an accelerated approval pathway for AMT-130, a gene therapy for Huntington's disease. The FDA agreed that data from ongoing studies are sufficient for regulatory filing, avoiding additional pre-submission studies. The therapy's safety profile is manageable, and uniQure plans to submit a biologics license application in 2025.

Global markets show mixed performance; U.S. indexes like S&P 500 and Nasdaq Composite reach record highs, while Russell 2000 Index declines. Growth stocks in IT and consumer discretionary sectors gain over 3%. High-growth tech stocks require consideration of innovation potential and financial health. Top 10 high-growth tech companies include Seojin SystemLtd, Yggdrazil Group, Ascelia Pharma, Waystream Holding, Medley, Alnylam Pharmaceuticals, TG Therapeutics, Fine M-TecLTD, Alkami Technology, and Initiator Pharma. Genmab A/S, Yubico AB, and Sharetronic Data Technology Co., Ltd. are highlighted for their strong growth and innovation in biotechnology, cybersecurity, and smart terminal technologies respectively.

Alnylam Pharmaceuticals launched a Phase 1 trial for ALN-HTT02, a huntingtin-lowering drug for Huntington’s disease, using RNA interference. The trial aims to assess safety and efficacy, with recruitment currently open in the UK and Canada. Participants, aged 25-70, will receive spinal injections of ALN-HTT02 or a placebo, with the option to switch to the drug after 12 months.

Clinical trials of ALN-HTT02, an siRNA-based drug targeting Huntington's disease, have begun at UCLH, offering hope for disease modification. The trial involves 54 participants and aims to analyze the drug's safety and efficacy, with potential to transform current treatment paradigms. Collaboration between pharmaceutical companies and research institutions is crucial for advancing such therapies.

Pozelimab and cemdisiran combination shows superior inhibition of terminal complement in PNH patients, achieving higher LDH control and normalization rates compared to ravulizumab, with potential for self-administration.

A clinical trial for Huntington's disease, using ALN-HTT02 to reduce damaging protein levels, has begun at UCLH. The trial aims to recruit 54 participants, offering either ALN-HTT02 or a placebo. Professor Sarah Tabrizi highlights the drug's potential to slow disease progression. Around 7,000 people in the UK live with Huntington's, an inherited condition with a 50% chance of transmission. Symptoms include memory problems, depression, and difficulty moving. The disease is fatal 10 to 30 years after symptoms appear.